Biogen's hemophilia B treatment meets study goals

--Biogen says one of two key studies on potential hemophilia treatments is positive

--Company plans U.S. application for hemophilia B in first half next year

--Results still expected this year for study on potential treatment for hemophilia A, which is a bigger market

(Updates with details on study, background throughout.)

Biogen Idec Inc. BIIB said Wednesday that an experimental treatment for hemophilia B, one form of a rare bleeding disorder, met key goals in a late-stage study while paving the way for a U.S. regulatory application next year.

The Weston, Mass., company has been developing treatments for the two main types of hemophilia that are designed to last longer than current treatments, meaning fewer intravenous infusions for patients. Data from the late-stage study on hemophilia A--the bigger of the two markets--isn't out yet, but is still expected before the end of this year.

The data on Biogen's potential hemophilia B treatment could push the company into competition with Pfizer Inc. PFE, which currently has the main drug in the roughly $1 billion global market for this version of the disease, according to Credit Suisse. The bigger, $5 billion hemophilia A market is led by Baxter International Inc. BAX.

According to Biogen, the "B-Long" study of 123 patients showed the treatment--known as factor IX--was effective in controlling and preventing bleeding and was generally well tolerated. No patients in the study developed so-called inhibitors that can require more treatments and greater cost to try and control bleeding.

Biogen released top-line data and expects to report further results at a future scientific meeting.

"The primary efficacy and safety objectives were met," and a Food and Drug Administration application is planned for the first half next year, the company said.

Biogen shares rose 1.8% to $155.57 in premarket trading. They are up 39% on the year through Tuesday, spurred by high expectations for the company's late-stage product pipeline, including the hemophilia treatments. Investors are also closely watching an oral multiple sclerosis drug now pending with regulators and a potential treatment for Lou Gehrig's disease.

Hemophilia is a typically inherited condition in which patients, usually males, lack or have low levels of a certain protein the blood needs to form clots that stop bleeding. Only about 18,000 people in the U.S. have been diagnosed with the disorder, according to the Cleveland Clinic.

There are two main types of the disease, hemophilia A and B. In treatments that can cost hundreds of thousands of dollars per year, patients require injections of replacement clotting factors--the treatment for hemophilia A is factor VIII and hemophilia B treatment is factor IX--made from either donated blood plasma or synthetic products that don't come from plasma.

Known as recombinants, these synthetic versions have come to dominate the market in developed countries. They were developed following an early 1980's tragedy in which hemophilia patients were broadly infected with HIV from contaminated products, and many later died. The nation's blood-product safety is "dramatically improved," according to the National Hemophilia Foundation.

The market for recombinant treatments is becoming more crowded, and multiple companies are working on longer-lasting treatments that could cut down on infusions, but Biogen has the lead. The company and its European distribution partner Swedish Orphan Biovitrum AB (SOBI.SK, BIOVF) plan to file an application in Europe after completing a required study in children under age 12.

The B-Long included male patients aged 12 or older. They were enrolled in four treatment arms, including weekly preventative treatment, individually tailored preventative treatment, treatments as needed to control bleeding and management for patients who required major surgery.

Over the last six months of the study, the median dosing interval was every 14 days for patients getting individualized preventative treatment, Biogen said.

Corrections & Amplifications

This item was corrected at 4:10 p.m. EDT to reflect that the treatment for hemophilia B is factor IX. The original version misstated the treatment as factor IV.

Corrections & Amplifications

This article was corrected Sept. 27, 2012 at 11:45 EDT to reflect that the treatment for hemophilia A is factor VIII, not factor XIII.

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"2nd UPDATE: Biogen's Hemophilia B Treatment Meets Key Goals in Study," published at 12:31 p.m. EDT, misstated that the hemophilia B treatment is factor IV in the 10th paragraph. The treatment is factor IX. The error also occurred in the ninth paragraph of an earlier update at 9:36 a.m. EDT.

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"2nd UPDATE: Biogen's Hemophilia B Treatment Meets Key Goals in Study," published Wednesday at 12:31 p.m. EDT, misstated the treatment for hemophilia A as factor XIII in the 10th paragraph. It is factor VIII. The error also occurred in an earlier update at 9:45 a.m. Wednesday.

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